Extract

Background and study aims: Alzheimer’s disease (AD) is the most common cause of dementia, affecting around 30 million individuals worldwide. It is caused by the abnormal build-up of various proteins in the brain to form what are known as amyloid plaques. The plaques are toxic to brain cells, and eventually cause their death, leading to the gradual decline in day-to-day memory and other mental functions. Serum amyloid P component (SAP) is a normal protein that occurs in everyone. It is produced in the liver and travels via the blood stream to reach other organs including the brain. Although only very small amounts of SAP enter the brain, it binds to the abnormal proteins in the brains of patients with AD. It forms part of the amyloid plaques and prevents them from breaking down. Therefore, preventing SAP from binding amyloid plaques may lead to faster breakdown of the amyloid plaques and so delay the progression of Alzheimer’s disease. There is also evidence that SAP directly damages brain cells and may contribute to the development of Alzheimer’s disease. Removal of SAP may reduce this damage to brain cells. A new drug has been developed, called CPHPC, which eliminates SAP almost completely from the blood and thereby stops SAP from reaching the brain. CPHPC may also remove the SAP already present in the brain. This may reduce the brain damage caused by the disease. The aim of this study is to explore the safety, tolerability and potential effectiveness of CPHPC in patients with mild AD. Who can participate? Patients aged 50-80 who have been diagnosed with mild AD. What does the study involve? Participants are randomly allocated to one of two groups. Those in the first group receive three injections per day of CPHPC into their tummy for 12 months. Those in the second group receive three injections per day of a placebo (dummy drug) into their tummy for 12 months. At the start of the study, over the 12 months of treatment, and one month later, participants in both groups undergo brain scanning to look at the effects of the treatment, as well as having blood and cerebrospinal fluid (the fluid found in the brain and spinal cord) samples taken for testing and completing assessments of their memory. What are the possible benefits and risks of participating? It is not known whether taking part in this study will benefit participants however those who receive the CPHPC may benefit from a reduced amount of SAP in the blood and removal of SAP from the brain throughout the period of treatment. There are no anticipated risks, however as this is a study looking at a new experimental drug there is a potential that patients may experience side effects. Where is the study run from? National Institute for Health Research (UK) When is study starting and how long is it expected to run for? August 2015 to August 2020 Who is funding the study? Education Endowment Foundation (UK) Who is the main contact? Mr Chinaza Ezirim [email protected]

Inclusion criteria

• Specialty: Dementias and neurodegeneration, Primary sub-specialty: Dementia; UKCRC code/ Disease: Neurological/ Demyelinating diseases of the central nervous system

Links

• isrctn